Market Newsdesk

September 9, 2021 7:33 am

FDA Grants Soligenix Orphan Drug Designation for the Treatment of T-cell Lymphoma

Extension of hypericin orphan designation beyond cutaneous T-cell lymphoma

PR Newswire

PRINCETON, N.J., Sept. 9, 2021 /PRNewswire/ — Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to the active ingredient hypericin for the treatment of T-cell lymphoma, extending the target population beyond cutaneous T-cell lymphoma (CTCL) as previously granted.

The U.S. Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. In addition to providing a seven year term of market exclusivity upon final FDA approval, orphan drug designation also positions Soligenix to be able to leverage a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees for the potential submission of a New Drug Application (NDA), and certain tax credits.

“The FDA’s decision to grant and expand our hypericin orphan drug designation beyond CTCL signifies an important step for Soligenix as we continue to advance the program toward NDA filing in the first half of 2022,” stated Christopher J. Schaber, PhD, President & Chief Executive Officer of Soligenix. “HyBryte™’s biologic activity was clearly demonstrated in the positive Phase 3, pivotal FLASH (Fluorescent Light Activated Synthetic Hypericin) study in patients suffering with early stage CTCL. The marketing exclusivity that this broadened orphan drug designation imparts adds significantly to the existing patent estate surrounding hypericin.”

About HyBryte™

HyBryte™ (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later which triggers apoptosis of the cell. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte™ has previously received orphan drug and fast track designations from the U.S. FDA, as well as orphan designation from the European Medicines Agency (EMA) and Promising Innovative Medicine (PIM) and “Innovation Passport” under the Innovative Licensing and Access Pathway (ILAP) from the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom for CTCL.

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte™ treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte™ achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte™ treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte™ treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte™ treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte™ treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte™ continued to be safe and well tolerated. Additional analyses also indicated that HyBryte™ is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their CTCL lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte™ throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte™ is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte™ continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. Its mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix.

About T-Cell Lymphoma

T-cell lymphomas can develop in lymphoid tissues such as the lymph nodes and spleen, or outside of lymphoid tissues (i.e., gastrointestinal tract, liver, nasal cavity, skin, and others). A similar lymphocyte called a natural killer (NK) cell shares many features with T cells. When NK cells become cancerous, the cancer is called NK or NK/T-cell lymphoma and is generally grouped with other T-cell lymphomas. T-cell lymphomas account for about seven percent of all NHLs in the U.S. according to the Surveillance, Epidemiology, and End Results (SEER) program. Each particular subtype of T-cell lymphoma is very uncommon. They can be aggressive (fast-growing) or indolent (slow-growing).

Lymphomas are often, but not always, named from a description of the normal cell that leads to cancer. There are three main categories of T-cell lymphomas: Peripheral T-cell lymphoma (PTCL), CTCL and those arising from immature T-cells or lymphoblatic lymphoma. Treatment of T-cell lymphomas is driven by the specific cancer subtype and the organs affected, and can range from skin directed therapies to systemic therapies to stem cell transplantation.

PTCL represents about 60% of all mature T-cell lymphomas and is characterized by a number of sub-types. The three most common subtypes include PTCL, Not Otherwise Specified (PTCLNOS, ~20% of all T-cell lymphomas), Anaplastic Large Cell Lymphoma (ALCL, ~11%) and Angioimmunoblastic T-Cell Lymphoma (AITL, ~7%). In general, PTCLs will include some degree of skin involvement, and some subtypes may be specifically related to latent viral infections (e.g., AITL). Involvement of peripheral organs only, such as skin or lymph nodes, is generally associated with a better prognosis.

CTCL accounts for about 3-4 percent of all NHL cases and usually affects adults. The term CTCL describes a group of typically indolent lymphomas that appear on, and are most often confined to, the skin. Mycosis fungoides, which appears as skin patches, plaques, or tumors, is the most common type of CTCL. Patches are usually flat, possibly scaly, and look like a rash; plaques are thicker, raised, usually itchy lesions that are often mistaken for eczema, psoriasis, or dermatitis; and tumors are raised bumps, which may or may not ulcerate. More than one type of lesion may be present at any time. Sézary syndrome is a less common form of CTCL that affects both the skin and blood. The most common symptoms are swollen lymph nodes and a red, very itchy rash that covers large portions of the body. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas. More information on CTCL can be accessed through the Cutaneous Lymphoma Foundation.

There are other rarer types of T-cell lymphoma, including those arising in patients after transplantation and immunosuppression, viral infection such as human T-lymphotropic virus type 1 (HTLV-1) and lymphoblastic lymphoma. For a more detailed description, please refer to the fact sheet provided by the Lymphoma Research Foundation.

About Soligenix, Inc.

Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (HyBryte™ or synthetic hypericin) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With a successful Phase 3 study completed, regulatory approval is being sought and commercialization activities for this product candidate are being advanced initially in the U.S. Development programs in this business segment also include our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn’s disease (SGX203) and acute radiation enteritis (SGX201).

Our Public Health Solutions business segment includes active development programs for RiVax^®, our ricin toxin vaccine candidate, and SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease, and our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax^®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA).

For further information regarding Soligenix, Inc., please visit the Company’s website at https://www.soligenix.com and follow us on LinkedIn and Twitter at @Soligenix_Inc.

This press release may contain forward-looking statements that reflect Soligenix, Inc.’s current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations and clinical trial enrollment. Statements that are not historical facts, such as “anticipates,” “estimates,” “believes,” “hopes,” “intends,” “plans,” “expects,” “goal,” “may,” “suggest,” “will,” “potential,” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, such as experienced with the COVID-19 outbreak. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the U.S. Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the U.S. Congress may not pass any legislation that would provide additional funding for the Project BioShield program. In addition, there can be no assurance as to the timing or success of any of its clinical/preclinical trials. Despite the statistically significant result achieved in the HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma, there can be no assurance that a marketing authorization from the FDA or EMA will be successful. Further, there can be no assurance that RiVax^® will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax^®. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission, including, but not limited to, Soligenix’s reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.

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SOURCE Soligenix, Inc.

September 9, 2021 7:33 am

MindMed to Attend 8th Annual “Mobile in Clinical Trials” Conference

PR Newswire

NEW YORK, Sept. 9, 2021 /PRNewswire/ — Mind Medicine (MindMed) Inc. (NASDAQ: MNMD), (NEO: MMED), (DE: MMQ) (the “Company”), a leading biotech company developing psychedelic-inspired therapies, is pleased to announce that its Chief Medical Officer, Daniel R. Karlin, MD MA, will co-chair the 8th annual Mobile in Clinical Trials Conference, which will be held virtually on September 27, 2021 and is a partner program to the 2021 DPharm Conference.

“I’ve always enjoyed co-chairing Mobile in Clinical Trials, together with my friend Michelle Shogren from Bayer,” said Dr. Karlin. “We would have preferred a hybrid option allowing for in person interactions but the safety of attendees comes first. Last year’s virtual format still enabled the fruitful exchange of the latest knowledge in the area of digital solutions, just as we had experienced during previous years of Mobile in Clinical Trials that took place in person.”

The 8th annual Mobile in Clinical Trials Conference will feature the latest examples of deploying remote digital tools in clinical studies and will offer solutions for making digital technology an integral part of clinical research. Mobile in Clinical Trials is the event most attended by digital R&D operations leaders who are seeking solutions to apply mobile-digital tools into clinical research. Each year at this Conference, participants present experience-based case studies and exchange fresh ideas to advance fit-for-purpose adoption and scale-up of remote digital solutions in the area of clinical research.

About MindMed

MindMed is a clinical-stage psychedelic medicine biotech company that seeks to discover, develop and deploy psychedelic-inspired medicines and therapies to address addiction and mental illness. The Company is assembling a compelling drug development pipeline of innovative treatments based on psychedelic substances including psilocybin, LSD, MDMA, DMT and an Ibogaine derivative, 18-MC. The MindMed executive team brings extensive biopharmaceutical experience to MindMed’s approach to developing the next generation of psychedelic-inspired medicines and therapies.

MindMed trades on the NASDAQ under the symbol MNMD and on the Canadian NEO Exchange under the symbol MMED. MindMed is also traded in Germany under the symbol MMQ.

Forward-Looking Statements

Certain statements in this news release related to the Company constitute “forward-looking information” within the meaning of applicable securities laws and are prospective in nature. Forward-looking information is not based on historical facts, but rather on current expectations and projections about future events and are therefore subject to risks and uncertainties which could cause actual results to differ materially from the future results expressed or implied by the forward-looking statements. These statements generally can be identified by the use of forward-looking words such as “will”, “may”, “should”, “could”, “intend”, “estimate”, “plan”, “anticipate”, “expect”, “believe”, “potential” or “continue”, or the negative thereof or similar variations. Forward-looking information in this news release include, but are not limited to, statements regarding MindMed’s attendance at the Mobile in Clinical Trials Conference, Conference program and topics and Conference attendees. Although the Company believes that the expectations reflected in such forward-looking information are reasonable, such information involves risks and uncertainties, and undue reliance should not be placed on such information, as unknown or unpredictable factors could have material adverse effects on future results, performance or achievements of the Company. There are numerous risks and uncertainties that could cause actual results and the Company’s plans and objectives to differ materially from those expressed in the forward-looking information, including history of negative cash flows; limited operating history; incurrence of future losses; availability of additional capital; lack of product revenue; compliance with laws and regulations; difficulty associated with research and development; risks associated with clinical trials or studies; heightened regulatory scrutiny; early stage product development; clinical trial risks; regulatory approval processes; novelty of the psychedelic inspired medicines industry; as well as those risk factors discussed or referred to herein and the risks described under the headings “Risk Factors” in the Company’s filings with the securities regulatory authorities in all provinces and territories of Canada which are available under the Company’s profile on SEDAR at www.sedar.com and with the U.S. Securities and Exchange Commission on EDGAR at www.sec.gov. Should one or more of these risks or uncertainties materialize, or should assumptions underlying the forward-looking information prove incorrect, actual results and future events could differ materially from those anticipated in such information. Although the Company has attempted to identify important risks, uncertainties and factors that could cause actual results to differ materially, there may be others that cause results not to be as anticipated, estimated or intended. These and all subsequent written and oral forward-looking information are based on estimates and opinions of management on the dates they are made and are expressly qualified in their entirety by this notice. Except as required by law, the Company does not intend and does not assume any obligation to update this forward-looking information.

Media Contact: [email protected]

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SOURCE Mind Medicine (MindMed) Inc.

September 9, 2021 7:33 am

Biohaven Spotlights Nurtec® ODT (rimegepant), the only dual-therapy indicated for both the acute and preventive treatment of migraine, at the 2021 International Headache Society – European Headache Federation Joint Congress

– Biohaven presenting 17 abstracts, including three late-breaking abstracts and four oral presentations, highlighting the efficacy and safety of Nurtec ODT as both an acute and preventive treatment of migraine

– Preventive effects of rimegepant evident as early as the first week of treatment and sustained after 12 weeks to be presented from Biohaven’s Phase 2/3 Study

– A patient preference study demonstrated that those who used rimegepant for one year, four in five preferred it to their previous migraine medications, seven in 10 were satisfied, and nine in 10 experienced clinical improvement relative to baseline

– Health economics and outcomes research posters highlight the association between migraine-related absenteeism, healthcare utilization and cost of care, and showcased improved quality of life with rimegepant

PR Newswire

NEW HAVEN, Conn., Sept. 9, 2021 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), today announced that 17 abstracts, including three late-breaking presentations and four oral presentations, will be shared at the 2021 International Headache Society – European Headache Federation Joint Congress virtually from September 8-12, 2021.

Biohaven will be presenting most recent data for its migraine portfolio including Nurtec® ODT (rimegepant) orally dissolving tablets 75 mg, the first and only calcitonin gene-related peptide (CGRP) receptor antagonist in an orally disintegrating tablet (ODT) approved by the FDA for the acute treatment of migraine and the preventive treatment of episodic migraine in adults; and intranasal zavegepant, the only intranasal CGRP receptor antagonist currently in late-stage clinical trials for the acute treatment of migraine.

The rimegepant studies include two late breaking abstracts and two oral presentations spotlighting the efficacy and safety data for rimegepant as an acute and preventive treatment of migraine. One poster showcases Phase 2/3 data for rimegepant and other highlights several health economics and outcomes research analyses including a late breaking oral lecture on time to monthly migraine days (MMDs) reduction.

Elyse Stock, MD, Chief Medical Officer of Biohaven commented, “Nurtec ODT has changed the treatment paradigm for migraine with an easy to use, oral CGRP antagonist that is now indicated for both the acute treatment and prevention of migraine. Our research efforts to help people with migraine are demonstrated by the breadth of posters presented at the 2021 International Headache Congress. Our focus remains strong on analyzing the burden of migraine and our health economics and outcomes research analysis spotlights important issues affecting those living with this debilitating disease.”

Notable highlights include:

Data from a Phase 2/3 clinical trial to evaluate the safety and efficacy of rimegepant as a preventive treatment of migraine compared to placebo demonstrated that oral rimegepant taken every other day showed rapid migraine preventive effects as early as the first week of dosing. Additionally, it was observed to be superior to placebo across both primary and secondary endpoints of the trial. These include ≥50% reduction in the mean number of moderate or severe MMDs during weeks 9-12 (49% vs 41%, p=0.0438) and change in the mean number of total MMDs during Weeks 1-12 (−4.3 vs −3.5, p=0.0099).
Results from an open label safety study of rimegepant demonstrated its preventive benefits when dosed on an as needed basis to treat migraine attacks, expressed as median time to 30% and 50% reduction in MMDs observed to decrease at 12 weeks and 32 weeks respectively. Additionally, the long-term use of rimegepant 75 mg was associated with reductions in MMDs without evidence of medication-related increases in headache frequency. A different analysis that evaluated the safety of rimegepant in those with cardiovascular (CV) risk showed that rimegepant dosed up to once daily for up to 1 year showed favorable safety and tolerability in adults with migraine with CV risk factors, including adults with moderate to high CV risk.
A long-term open label safety study assessed patient preference, satisfaction, and improved clinical global impression of change (CGI-C) with rimegepant 75 mg for the acute treatment of migraine. Data showed that at week 24, 78.7% of subjects preferred rimegepant over their previous migraine medications, 89.4% of subjects were satisfied with rimegepant, and 88.8% of subjects were considered improved since study entry on the CGI-C scale. Among individuals using rimegepant as an acute treatment for one year, four in five preferred rimegepant to their previous migraine medications, seven in 10 were satisfied with rimegepant, and nine in 10 experienced clinical improvement relative to baseline.
A U.S.-based real world longitudinal analysis showed that migraine-related absenteeism was common, affecting more than two-thirds of the analyzed cohort and was associated with higher medical and pharmaceutical claims and healthcare costs. The analysis evaluated migraine-related absenteeism and its association with healthcare utilization and cost among adults with migraine.
A Phase 2/3 dose-ranging study evaluating the safety, efficacy and tolerability of intranasal zavegepant for the acute treatment of migraine demonstrated that zavegepant 10 mg and 20 mg were superior to placebo on the coprimary endpoints of pain freedom [placebo: 15.5%; 5 mg: 19.6% (p=0.1214); 10 mg: 22.5% (p=0.0113); 20 mg: 23.1% (p=0.0055)] and most bothersome symptom [placebo: 33.7%; 5 mg: 39.0% (p=0.1162); 10 mg: 41.9% (p=0.0155); 20 mg: 42.5% (p=0.0094)] at two hours post dose. The most common (>5%) adverse events (AEs) with zavegepant were altered taste (13.5%-16.1% vs 3.5% with placebo) and nasal discomfort (1.3%-5.2% vs 0.2% with placebo).

The complete list of accepted abstract titles is below and full presentations will be available to registered participants on the Congress website beginning September 8, 2021.

Oral Presentations:

(Late-breaker) Acute Treatment with Rimegepant 75 mg Confers Long Term Improvements in Median Time to 30% and 50% Reductions in Monthly Migraine Days – Post Hoc Results from an Open Label Safety Study (BHV3000-201)
Onset of Migraine Preventive Effects With Rimegepant in a Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Trial
A Phase 2/3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rimegepant for the Preventive Treatment of Migraine
Migraine-Related Absenteeism is Associated With Total Healthcare and Pharmaceutical Costs — A US-Based Real World Longitudinal Analysis

Poster Presentations:

(Late-breaker) Understanding the Patient Experience and Disease Burden of Migraine: a Qualitative Interview Study
(Late-breaker) Effect of Strong P-gp and BCRP Inhibition, Using Cyclosporine and Quinidine as Probes, on the Pharmacokinetics of Oral Rimegepant 75 mg in Healthy Subjects
Oral Rimegepant 75 mg is Safe and Well Tolerated in Adults With Migraine and Cardiovascular Risk Factors: Results of a Multicenter, Long-Term, Open-Label Safety Study
Acute Treatment with Oral Rimegepant 75 mg Reduces Migraine-Related Disability in Adults With and Without a History of Triptan Treatment Failure: Results from a One Year, Open-Label Safety Study
Acute Treatment of Migraine With Rimegepant Improves Health Related Quality of Life in Adults With a History of Triptan Treatment Failure: Results from a Long-Term, Open-Label Safety Study
Long-term Use of Rimegepant 75 mg for the Acute Treatment of Migraine Reduces Use of Analgesics and Antiemetics
Rimegepant for the Acute Treatment of Migraine: Subgroup Analyses From 3 Phase 3 Clinical Trials by Number of Triptans Previously Tried and Failed
Patient Preference, Satisfaction, and Improved Clinical Global Impression of Change with Rimegepant 75 mg for the Acute Treatment of Migraine: Results from a Long-Term Open-Label Safety Study
Rimegepant 75 mg for the Acute Treatment of Migraine in Adults With Frequent Migraine: Long-Term Safety and Clinical Improvement Versus Baseline
Rimegepant Versus Atogepant and Monoclonal Antibody Treatments for the Prevention of Migraine: A Systematic Literature Review and Network Meta-analysis
Monthly Migraine Days, Tablet Utilization, and Quality of Life Associated with Rimegepant – Post Hoc Results from an Open Label Safety Study (BHV3000-201)
Intranasal Zavegepant is Effective and Well Tolerated for the Acute Treatment of Migraine: A Phase 2/3 Dose-Ranging Clinical Trial
Economic Modeling of Migraine Prevention Therapies: Considerations for Current and Emerging Therapies

About Nurtec ODT
NURTEC ODT (rimegepant) is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in a quick-dissolve ODT formulation that is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine in adults. The activity of the neuropeptide CGRP is thought to play a causal role in migraine pathophysiology. NURTEC ODT is a CGRP receptor antagonist that works by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the CGRP neuropeptide. The recommended dose of NURTEC ODT is 75 mg, taken as needed, up to once daily to treat or every other day to help prevent migraine attacks. For more information about NURTEC ODT, visit www.nurtec.com. The most common adverse reaction was nausea and abdominal pain/indigestion. Avoid concomitant administration of NURTEC ODT with strong inhibitors of CYP3A4, strong or moderate inducers of CYP3A or inhibitors of P-gp or BCRP. Avoid another dose of NURTEC ODT within 48 hours when it is administered with moderate inhibitors of CYP3A4.

Indication
NURTEC ODT orally disintegrating tablets is a prescription medicine that is used to treat migraine in adults. It is for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine. It is not known if NURTEC ODT is safe and effective in children.

Important Safety Information
Do not take NURTEC ODT if you are allergic to NURTEC ODT (rimegepant) or any of its ingredients.

Before you take NURTEC ODT, tell your healthcare provider (HCP) about all your medical conditions, including if you:

have liver problems,
have kidney problems,
are pregnant or plan to become pregnant,
breastfeeding or plan to breastfeed.

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

NURTEC ODT may cause serious side effects including allergic reactions, including trouble breathing and rash. This can happen days after you take NURTEC ODT. Call your HCP or get emergency help right away if you have swelling of the face, mouth, tongue, or throat or trouble breathing. This occurred in less than 1% of patients treated with NURTEC ODT.

The most common side effects of NURTEC ODT were nausea (2.7%) and stomach pain/indigestion (2.4%). These are not the only possible side effects of NURTEC ODT. Tell your HCP if you have any side effects.

You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088 or report side effects to Biohaven at 1-833-4NURTEC.

Please click here for full Prescribing Information and Patient Information.

About Zavegepant
Zavegepant is a third generation, high affinity, selective and structurally unique, small molecule CGRP receptor antagonist from Biohaven’s NOJECTION™ Migraine Platform and the only CGRP receptor antagonist in clinical development with both intranasal and oral formulations. The efficacy and safety profile of intranasal zavegepant for the acute treatment of migraine, as compared to placebo, was shown in a randomized controlled Phase 2/3 dose-ranging trial with a total of over 1000 patients who received zavegepant. In this study, zavegepant showed statistical superiority to placebo on the coprimary endpoints of 2 hour freedom from pain and freedom from a patients’ most bothersome symptom (either nausea, photophobia or phonophobia). Following successful end of Phase 2 interactions with FDA (clinical and nonclinical), zavegepant is advancing to Phase 3 for the acute treatment of migraine in adults. For more information, visit https://www.biohavenpharma.com/science-pipeline/cgrp/bhv-3500.

About Biohaven
Biohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. Biohaven’s neuroinnovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine and CGRP-mediated neuroimmune/neuroinflammatory diseases; glutamate modulation for obsessive-compulsive disorder, Alzheimer’s disease, and spinocerebellar ataxia; and myeloperoxidase (MPO) inhibition for multiple system atrophy and amyotrophic lateral sclerosis. More information about Biohaven is available at www.biohavenpharma.com.

Forward-looking Statement
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including “believe”, “continue”, “may”, “will” and similar expressions, are intended to identify forward-looking statements. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of Biohaven’s management about NURTEC ODT as an acute treatment for patients with migraine and preventive treatment for migraine. Factors that could affect these forward-looking statements include those related to: Biohaven’s ability to effectively commercialize NURTEC ODT, delays or problems in the supply or manufacture of NURTEC ODT, complying with applicable U.S. regulatory requirements, the expected timing, commencement and outcomes of Biohaven’s planned and ongoing clinical trials, the timing of planned interactions and filings with the FDA, the timing and outcome of expected regulatory filings, the potential commercialization of Biohaven’s product candidates, the potential for Biohaven’s product candidates to be first in class or best in class therapies and the effectiveness and safety of Biohaven’s product candidates. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by forward-looking statements. Additional important factors to be considered in connection with forward-looking statements are described in the “Risk Factors” section of Biohaven’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission on March 1, 2021, and in Biohaven’s subsequent filings with the Securities and Exchange Commission. The forward-looking statements are made as of this date and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

NURTEC and NURTEC ODT are registered trademarks of Biohaven Pharmaceutical Ireland DAC. Neuroinnovation is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

Biohaven Contact
Dr. Vlad Coric
Chief Executive Officer
[email protected]

Media Contact
Mike Beyer
Sam Brown Inc.
[email protected]
312-961-2502

View original content to download multimedia:https://www.prnewswire.com/news-releases/biohaven-spotlights-nurtec-odt-rimegepant-the-only-dual-therapy-indicated-for-both-the-acute-and-preventive-treatment-of-migraine-at-the-2021-international-headache-society–european-headache-federation-joint-congress-301372169.html

SOURCE Biohaven Pharmaceutical Holding Company Ltd.

September 9, 2021 7:33 am

Amesite Inc. to Sponsor and Present at Sequire EdTech Event on September 13th

Presentation on Monday, September 13th, 2021 at 12:00 PM ET

PR Newswire

DETROIT, Sept. 9, 2021 /PRNewswire/ — Amesite Inc. (Nasdaq: AMST), an artificial intelligence software company providing advanced A.I. powered online learning ecosystems for business and higher education, announced today that it will be presenting virtually at the upcoming Sequire EdTech Conference event on Monday, September 13th at 12:00 PM ET. Dr. Ann Marie Sastry, Founder and CEO of Amesite Inc, will be giving the presentation.

“I look forward to sharing our story with the investment community at the Sequire EdTech Conference,” said Dr. Sastry. “Online learning has become integral, both within our education system and in the business community, and this trend shows no signs of abating. Businesses need to train people, to enable them to adapt, be more productive, and drive revenue – and Amesite, as a Microsoft Partner, is well-positioned to help.”

Event: Amesite Inc. Presentation at the Sequire EdTech Conference
Date: Monday, September 13th, 2021
Time: 12:00 – 12:25 PM ET

Event: Higher Education and the Future of Work with Ann Marie Sastry, Mary Juhas (Associate VP at Ohio State University) & Barbie Brewer (Chief People Officer at ClickUp)
Date: Monday, September 13th, 2021
Time: 1:30 – 1:55 PM ET

Event: The Coming Decades of Disruptive Innovation in Education with Ann Marie Sastry & Jonathan Satchell (Chief Executive at Learning Technologies Group plc)
Date: Monday, September 13th, 2021
Time: 2:00 – 2:25 PM ET

Summary of Sequire EdTech Conference

The Sequire EdTech Conference is a must-see event featuring a full day of EdTech presentations from publicly-traded companies, as well as keynotes and panels with industry experts on the future of work and education.

About Amesite Inc.

Amesite is an ed-tech, SaaS company with the most advanced artificial intelligence driven online learning platform in the industry, providing both content creation and a best-in-class infrastructure for the multibillion-dollar online learning markets in business and education. For more information, visit https://amesite.com.

Investor Relations Contact:

RedChip Companies Inc.
Dave Gentry, CEO
[email protected]
1-800-RED-CHIP (733-2447)
(or) 407-491-4498

View original content to download multimedia:https://www.prnewswire.com/news-releases/amesite-inc-to-sponsor-and-present-at-sequire-edtech-event-on-september-13th-301371798.html

SOURCE Amesite

September 9, 2021 7:33 am

Thryv Named Easiest to Use Small Business Software in G2 Fall Report

Dallas, Sept. 09, 2021 (GLOBE NEWSWIRE) — Thryv, Inc. (NASDAQ:THRY), the provider of Thryv ^® software and Hub by Thryv™, the end-to-end customer experience platform built for growing small businesses and emerging franchises, has once again received multiple awards in the G2 Fall Report.

G2, an online marketplace where software shoppers can discover and review technology, has ranked Thryv a Leader for Small Businesses for two years straight (eight consecutive quarterly reports). G2’s awards are determined by detailed reviews written by verified software users.

In addition to being named a G2 Leader for Small Businesses and Overall, G2 Fall Report 2021 Leadership Awards for Thryv also include:

New this quarter, Thryv wins for Fastest Implementation. This means that Thryv has the shortest go-live time in its category, amongst all users.
For the sixth straight quarter, users say Thryv has the Easiest Set-up for Small Businesses and Overall.
Also, for the sixth quarter in a row, Small Business users recognized that Thryv has the Easiest Admin.
Making it the fifth quarter in a row, Thryv has the Highest User Adoption for Small Businesses. New this quarter, Thryv also wins for Highest User Adoption Overall.
Also, for the fifth straight quarter, Thryv Small Business users are Most Likely to Recommend Thryv.
For the fourth straight quarter, Thryv was named a Leader for Overall Best Support.

“Thryv is definitely an essential tool for any business,” said Raquel Stewart, from Paradise Roofing in Gastonia, N.C. “I can now do everything from one place. Customer files, documents, estimates, invoices and payments all together. It’s great.”

New for the 2021 G2 Fall Report, Thryv moved up 9 positions into the Top 20 CRM platforms, in the CRM Implementation Index: Small Business. With easier setup, faster implementation time and higher user adoption, Thryv surpassed competitors such as Keap and Zoho CRM in this critical category.

“When Thryv users recognize that our platform is faster and easier to set up, and they recommend it to their peers, that’s when we know we’ve created successful solutions that truly help small business owners,” said Ryan Cantor, Thryv’s VP of Product and Marketing.

Thryv’s end-to-end customer experience platform centralizes the tools small businesses use every day, such as estimates, invoicing, payment requests and reminders, and payment processing with ThryvPay^SM, Google My Business integration, a verticalized CRM tailored to various industries, email and SMS text marketing automation, online appointment scheduling, social media and online review management and more. Hub by Thryv offers emerging franchises the ability to scale their multi-location business with a turn-key franchise business software that provides oversight of every location, all in one place.

For more information, visit Thryv.com.

About Thryv Holdings, Inc.

The company owns the easy-to-use Thryv® end-to-end customer experience software built for small business that helps over 40,000 SaaS clients with the daily demands of running a business. With Thryv, they can get the job, manage the job and get credit. Thryv’s award-winning platform provides modernized business functions, allowing small-to-medium-sized businesses (SMBs) to reach more customers, stay organized, get paid faster and generate reviews. These include building a digital customer database, automated marketing through email and text, updating business listings across the internet, scheduling online appointments, sending notifications and reminders, managing ratings and reviews, generating estimates and invoices and processing payments.

Thryv supports franchise operators and multi-location business owners with Hub by Thryv™, a software console that enables business managers to oversee their operations using the Thryv software.

Thryv also connects local businesses to consumer services through our search, display and social media management products, our print directories featuring The Real Yellow Pages® tagline, and our local search portals, which operate under the DexKnows.com®, Superpages.com® and Yellowpages.com URLs and reach some 35 million monthly visitors. For more information about the company, visit thryv.com.

Thryv delivers business services to more than 400,000 SMBs globally that enable them to compete and win in today’s economy.

Thryv acquired Sensis, Australia’s leading digital, marketing and directory services provider, which helps Australians connect and engage through its leading platforms, digital consumer businesses (Yellow™, White Pages™, TrueLocal™ and Whereis™), search engine marketing and optimization services, website products, social, data and mapping solutions, and through its digital agency Found™. Sensis is also Australia’s largest print directory publisher including the Yellow Pages™ and White Pages™.

Headquartered in Melbourne, Sensis has a sales presence in all states and territories across Australia.

Learn more about Thryv on LinkedIn and Medium.

Media Contact:

Paige Blankenship

Thryv, Inc.

214.392.9609

[email protected]

Investor Contact:

Cameron Lessard

Thryv, Inc.

214.773.7022

[email protected] 

KJ Christopher

Thryv, Inc.

972.453.7068

[email protected]

###

Paige Blankenship
Thryv, Inc.
2143929609
[email protected]

September 9, 2021 7:33 am

Vera Therapeutics Hosting Key Opinion Leader Webinar on IgA Nephropathy

SOUTH SAN FRANCISCO, Calif., Sept. 09, 2021 (GLOBE NEWSWIRE) — Vera Therapeutics, Inc. (Nasdaq: VERA), a clinical-stage biotechnology company focused on developing treatments for immunological diseases that improve patients’ lives, announced today that it is scheduled to host a key opinion leader (KOL) webinar on IgA Nephropathy (IgAN or Berger’s Disease) at 12:00 p.m. ET on Monday, September 20, 2021.

The event will feature presentations by KOLs Richard Lafayette M.D., Stanford University Medical Center, and Jonathan Barratt, Ph.D., FRCP, University of Leicester. Dr. Lafayette will discuss the rationale for targeting B lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL) in IgAN. Dr. Barratt will discuss data from trials targeting BLyS and APRIL in IgAN patients, including the Phase 2a JANUS study of atacicept, which evaluated the efficacy and safety of atacicept in IgAN. Both KOLs will discuss the potential for Vera’s atacicept as a treatment option. Atacicept is a recombinant fusion protein that acts as a dual inhibitor of cytokines BLyS and APRIL, which targets B cells and plasma cells, as well as reduces disease-causing autoantibodies.

Dr. Lafayette and Dr. Barratt will be available to answer questions following their formal presentations.

To register for the webinar, please click here.

KOL Biographies

Richard Lafayette, M.D., is a professor of Medicine (Nephrology) at the Stanford University Medical Center. Dr. Lafayette completed his medical education at New York Medical College and went on to complete his residency at the Long Island Jewish Medical Center, and his fellowship at Stanford University School of Medicine. He is board-certified in Internal Medicine and Nephrology.

Dr. Lafayette served as the Associate Chair of the Stanford University Department of Medicine from 2002 to 2007, the Clinical Chief of Nephrology at Stanford University from 1999 to 2012, and currently serves as the Director of the Stanford Glomerular Disease Center since 2010. He was honored in America’s Top Doctors, Best Doctors from 2004 to 2018, and received America’s Top Doctors Award, Castle Connolly Medical Ltd. from 2014 to 2022. Dr. Lafayette has been part of the following boards and professional organizations: Editorial Board, Kidney News, American Society of Nephrology (ASN) (2010 to 2021); Member, Glomerular Disease Advisory Committee, American Society of Nephrology (2013 to 2017); and, Member (ex-officio), Communications Committee, American Society of Nephrology (2015 to Present).

Jonathan Barratt, Ph.D., FRCP, leads the Renal Research Group within the College of Life Sciences, University of Leicester. Dr. Barratt’s research is focused on a bench to bedside approach to improving our understanding of the pathogenesis of IgAN, a common global cause of kidney failure. He is the IgAN Rare Disease Group lead for the United Kingdom National Registry of Rare Kidney Diseases (RaDaR), and a member of the steering committee for the International IgAN Network. He works closely with pharmaceutical companies interested in new treatments for IgAN, is chief investigator for a number of international randomized controlled Phase 2 and 3 clinical trials in IgAN, and was a member of the U.S. Food and Drug Administration and ASN Kidney Health Initiative: Identifying Surrogate Endpoints for Clinical Trials in IgAN Workgroup.

About Atacicept

Atacicept is a novel, disease-modifying fully humanized fusion protein that is a dual inhibitor of the cytokines B lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL). These cytokines are members of the tumor necrosis factor family that promote B-cell survival and autoantibody production associated with immunologic diseases, including IgA nephropathy (IgAN), also known as Berger’s disease, and systemic lupus erythematosus. Vera believes that atacicept has the potential to be the best-in-class and the leading B cell-targeted therapy for IgAN. Atacicept has been well tolerated and has been used in clinical trials of more than 1,500 patients to date. In a clinical trial of IgAN patients, data show atacicept is the first known molecule to substantially reduce galactose-deficient immunoglobulin A (Gd-IgA1).

About Vera

Vera Therapeutics is a clinical-stage biotechnology company focused on developing treatments for immunological diseases that improve patients’ lives. Vera’s lead product candidate is atacicept, a fusion protein self-administered as a subcutaneous injection once weekly that blocks both B lymphocyte stimulator (BLyS) and a proliferation-inducing ligand (APRIL), which stimulate B cells and plasma cells to produce autoantibodies contributing to certain autoimmune diseases. In addition, Vera is evaluating additional diseases where the reduction of autoantibodies by atacicept may prove medically useful, including lupus nephritis, a severe renal manifestation of systemic lupus erythematosus. For more information, please visit www.veratx.com.

Forward-looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential of atacicept. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will”, “anticipates,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Vera’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Vera’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Vera’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Vera undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Contacts

Investor Contact:
[email protected]

Media Contact:
Greig Communications, Inc.
Kathy Vincent
(310) 403-8951
[email protected]

September 9, 2021 7:33 am

Matrix Service Company To Present at Upcoming D.A. Davidson and Sidoti Virtual Investor Conferences

TULSA, Okla., Sept. 09, 2021 (GLOBE NEWSWIRE) — Matrix Service Company (Nasdaq: MTRX) announced today that President and Chief Executive Officer John R. Hewitt and Vice President and Chief Financial Officer Kevin Cavanah will be presenting at the D.A. Davidson 20th Annual Diversified Industrials & Services Conference on September 22, 2021 at 5pm Eastern Standard Time and at the Sidoti Fall Small Cap Investor Conference on September 23, 2021 at 10:00 a.m. Eastern Standard Time. One-on-one meetings with management are available during both conferences with prior notice and may be scheduled through the conferences or by contacting Matrix Service Company at [email protected].

About Matrix Service Company

Matrix Service Company (Nasdaq: MTRX), through its subsidiaries, is a leading North American industrial engineering and construction contractor headquartered in Tulsa, Oklahoma with offices located throughout the United States and Canada, as well as Sydney, Australia and Seoul, South Korea.

The Company reports its financial results in three key operating segments: Utility and Power Infrastructure, Process and Industrial Facilities, and Storage and Terminal Solutions.

With a focus on sustainability, building strong Environment, Social and Governance (ESG) practices, and living our core values, Matrix ranks among the Top Contractors by Engineering-News Record, was recognized for its Board diversification by 2020 Women on Boards, is an active signatory to CEO Action for Diversity and Inclusion, and is consistently recognized as a Great Place to Work®. To learn more about Matrix Service Company, visit matrixservicecompany.com

For more information about Matrix, please contact:

Kevin S. Cavanah	Kellie Smythe
Matrix Service Company	Matrix Service Company
Vice President and CFO	Senior Director, Investor Relations
T: 918-838-8822	T: 918-359-8267
Email: [email protected]	Email: [email protected]

This release contains forward-looking statements that are made in reliance upon the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are generally accompanied by words such as “anticipate,” “continues,” “expect,” “forecast,” “outlook,” “believe,” “estimate,” “should” and “will” and words of similar effect that convey future meaning, concerning the Company’s operations, economic performance and management’s best judgment as to what may occur in the future. Future events involve risks and uncertainties that may cause actual results to differ materially from those we currently anticipate. The actual results for the current and future periods and other corporate developments will depend upon a number of economic, competitive and other influences, including those factors discussed in the “Risk Factors” and “Forward Looking Statements” sections and elsewhere in the Company’s reports and filings made from time to time with the Securities and Exchange Commission. Many of these risks and uncertainties are beyond the control of the Company, and any one of which, or a combination of which, could materially and adversely affect the results of the Company’s operations and its financial condition. We undertake no obligation to update information contained in this release.

September 9, 2021 7:33 am

Centessa Pharmaceuticals Announces Positive Topline Data from Proof-of-Concept Study of SerpinPC in Severe Hemophilia A and B Patients Not on Prophylaxis

~ 88% reduction in median Annualized Bleeding Rate (ABR) for all bleeds and 94% reduction in median ABR for spontaneous joint bleeds in highest dose tested ~

~ SerpinPC observed to be well-tolerated ~

~ Company has initiated planning for global registrational program ~

~ Conference call and webcast scheduled for today at 8:30 a.m. EDT ~

CAMBRIDGE, Mass. and LONDON, Sept. 09, 2021 (GLOBE NEWSWIRE) — Centessa Pharmaceuticals plc (“Company”) (Nasdaq: CNTA), together with subsidiary ApcinteX Limited (“ApcinteX”), today announced positive topline results from the Phase 2a part of AP-0101, the six-month repeat dose portion of its ongoing first-in-human proof-of-concept study evaluating SerpinPC in severe hemophilia A and B patients.

AP-0101 is a Phase 1/2a proof-of-concept study evaluating SerpinPC, an inhibitor of activated protein C (“APC”), in 23 male subjects with either severe hemophilia A or B who were not on prophylaxis.¹ The Phase 2a part of the study assessed the safety, tolerability and pharmacokinetics across three dose cohorts (0.3 mg/kg, 0.6 mg/kg and 1.2 mg/kg) of SerpinPC administered as a subcutaneous (SC) injection every 4 weeks over a 24-week period (6 total doses). Reduction in the annualized bleeding rates (ABRs) were exploratory outcomes. Although eligible, none of the patients in the study had inhibitors.

SerpinPC was well-tolerated. As previously disclosed, one subject with a history of a skin disorder discontinued treatment on SerpinPC due to an injection site reaction. No other SerpinPC-related adverse events have been recorded. There was no reported sustained elevation in D-dimer, a sensitive measure of excess thrombin generation, throughout the 24-week study. Two subjects had anti-drug antibodies and remained on treatment without apparent impact on ABRs.

In the highest dose cohort, SerpinPC reduced the self-reported all bleeds ABR by 88% during the last 12 weeks of treatment (pre-specified primary assessment period) as compared to the all bleeds ABR prospectively measured during the pre-exposure observation period. In the highest dose cohort, five out of eight subjects had zero or one bleed during the 12-week pre-specified primary assessment period. Self-reported spontaneous joint bleeds ABR was reduced by 94% in the highest dose cohort. ABR reductions were similar between patients with either hemophilia A or hemophilia B.

	Dose Tested
Exploratory Efficacy Endpoints	0.3 mg/kg n=7	0.6 mg/kg n=7	1.2 mg/kg n=8
All Bleeds ABR (median percent change)	-80% p=0.016	-70% p=0.031	-88% p=0.016
Spontaneous Joint Bleeds ABR (median percent change)	-76% p=0.016	-69% p=0.031	-94% p=0.023
Above analyses compared last 12 weeks of treatment (pre-specified primary assessment period) to pre-exposure baseline measures. Bleeding events were self-reported. p-values presented are based on small numbers and are exploratory in nature.

The median number of target joints (joint with >3 bleeds in any 6-month period) was reduced to zero at the end of the study from a pre-exposure baseline of 2.5. All subjects had target joints at the start of the study and 15 subjects had zero target joints at the end of the study.

All 22 patients who completed the Phase 2a portion of the study have elected to enroll into the 48-week open label extension (“OLE”) portion of the study in which a single flat 60 mg subcutaneous dose of SerpinPC will be administered every 4 weeks over a period of 48 weeks (13 doses total). Centessa expects to report results from the OLE portion of this study in the second half of 2022.

“The compelling reduction in bleeds and continued tolerability that we observed in both hemophilia A and hemophilia B patients in this proof-of-concept study are very encouraging, and we are eager to move SerpinPC into a global development plan aimed at pursuing one or more registrations. We see broad utility of SerpinPC across the hemophilia landscape and will seek the most rapid path to bring this potential subcutaneous therapy to hemophilia patients,” said Antoine Yver, M.D., M.Sc., Chief Medical Officer of Centessa Pharmaceuticals.

“The results of this Phase 2a study of SerpinPC continue to show an excellent tolerability profile for this molecule, and the exploratory efficacy results seen in this study of severe hemophilia A and B patients are also very promising. A safe, subcutaneous, prophylaxis option for both hemophilia A and B patients would be an important addition to our treatment choices,” said David Lillicrap, M.D., Professor of Pathology and Molecular Medicine at Queen’s University, Kingston, Ontario, Canada and previously a World Federation of Hemophilia Advisory Board member.

^{¹ Clinicaltrials.gov identifier: NCT04073498 (https://clinicaltrials.gov/ct2/show/NCT04073498)}

Conference Call and Webcast

Centessa Pharmaceuticals will host a webcast and conference call today, September 9, 2021, at 8:30 a.m. EDT to discuss topline data from the proof-of-concept trial. To access the audio webcast with slides, please visit the “Events & Publications” page in the Investors & Media section of the Company’s website at https://investors.centessa.com/events-presentations. The call can also be accessed by dialing (855) 493-3565 (domestic) or (929) 517-9002 (international) with conference ID 8459296. An archive of today’s webcast will be available on the Company’s website.

About Centessa Pharmaceuticals

Centessa Pharmaceuticals plc aims to bring impactful new medicines to patients by combining the strengths of an asset-centric model with the benefits of scale and diversification typical of larger R&D organizations. The asset-centric model refers to a highly specialized, singular-focused company that is led by a team of well-recognized subject matter experts. Centessa’s asset-centric companies’ programs range from discovery-stage to late-stage development and include diverse therapeutic areas such as oncology, hematology, immunology/inflammation, neuroscience, hepatology, pulmonology and nephrology. For more information, visit www.centessa.com.

About ApcinteX Limited

ApcinteX Limited is focused on developing SerpinPC for the treatment of hemophilia A and hemophilia B. Hemophilia is a rare bleeding disorder that is caused by a deficiency of thrombin generation upon vascular damage.

About SerpinPC

SerpinPC, a biologic based on the serpin family of proteins, is designed to allow more thrombin to be generated by inhibiting activated protein C (APC) thus rebalancing coagulation in hemophilia patients. SerpinPC has the potential to treat all types of hemophilia regardless of severity or inhibitor status, and may also prevent bleeding associated with other bleeding disorders.

About AP-0101

AP-0101 is an ongoing Phase 1/2a open-label clinical trial to investigate the safety, tolerability and pharmacokinetics of intravenous and subcutaneous doses of SerpinPC in healthy male volunteers and male persons with severe hemophilia (https://clinicaltrials.gov/ct2/show/NCT04073498).

About Hemophilia A (HA) and Hemophilia B (HB)

HA and HB are X-linked genetic disorders affecting one in 5,000 and one in 20,000 live male births, respectively, resulting in spontaneous internal bleeding that can be life-threatening. More than 70% of bleeds occur into joints (hemarthrosis) causing chronic joint damage (arthropathy) with musculoskeletal destruction. The bleeding associated with these disorders is the result of a defect or deficiency in factor VIII (in the case of HA) or factor IX (in the case of HB), the two components of the intrinsic tenase complex.

Normal blood coagulation (hemostasis) is a crucial part of the physiological response to tissue damage. When blood components come into contact with extravascular cells and proteins, platelets accumulate and ultimately lead to the formation of thrombin, the effector enzyme of blood coagulation. Prothrombinase activity is required for the rapid, localized production of thrombin needed for adequate blood clotting. Prothrombinase is continuously degraded by APC, which is present in the circulation at low concentrations. In the setting of deficient intrinsic tenase activity (hemophilia), the natural anticoagulant activity of the circulating APC results in insufficient prothrombinase activity for normal blood clotting.

Forward Looking Statements
This press release contains forward-looking statements. These statements may be identified by words such as “believe,” “anticipate,” “plan,” “expect,” “intend,” “will,” “may,” “goal,” “project,” “estimate,” “potential,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These statements include discussions of the open label extension study of SerpinPC and its design and conduct; plans for continued development of SerpinPC, including our global development plan and registrational path for this candidate; our expectations with respect to the treatment paradigm for hemophilia A and B; our ability to deliver impactful medicines to patients; the ability of our key executives to drive execution of our portfolio of programs; our asset-centric business model and the intended advantages and benefits thereof; research and clinical development plans; the scope, progress, results and costs of developing our product candidates or any other future product candidates; strategy; regulatory matters, including the timing and likelihood of success of obtaining approvals to initiate or continue clinical trials or market any products; market size and opportunity; and our ability to complete certain milestones.

Any forward-looking statements in this press release are based on our current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the safety, tolerability and efficacy profile of SerpinPC observed to date may change adversely in future clinical trials, ongoing analyses of trial data or subsequent to commercialization; foreign regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; risks inherent in developing products and technologies; risks related to our ability to protect and maintain our intellectual property position; business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about the Company; our ability to obtain adequate financing to fund our planned clinical trials and other expenses; trends in the industry; the legal and regulatory framework for the industry; future expenditures risks related to our asset-centric corporate model; the risk that any one or more of our product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and risks related to the COVID-19 pandemic including the effects of the Delta variant. These and other risks concerning our programs and operations are described in additional detail in our most recent Form 10-Q, which is on file with the SEC and available on the SEC’s website at www.sec.gov. We operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on our current expectations, and speak only as of the date hereof. We explicitly disclaim any obligation to update any forward-looking statements except to the extent required by law.

Contacts:

Investor Contact:	Media Contacts:
Jennifer Porcelli, Head of Investor Relations	US
Centessa Pharmaceuticals	Dan Budwick, 1AB
[email protected]	[email protected]

	*UK/Greater Europe*
	Mary Clark, Optimum Strategic Communications
	[email protected]

	*Switzerland*
	Marcus Veith, VEITHing Spirit
	[email protected]
	M: +41 79 20 75 111

September 9, 2021 7:33 am

InflaRx to Present at the H.C. Wainwright 23rd Annual Global Investment Conference

JENA, Germany, Sept. 09, 2021 (GLOBE NEWSWIRE) — InflaRx (Nasdaq: IFRX), a clinical-stage biopharmaceutical company developing anti-inflammatory therapeutics by targeting the complement system, announces that Prof. Niels C. Riedemann, Chief Executive Officer and Founder, will present a company overview at the H.C. Wainwright 23^rd Annual Global Investment Conference, which is being held virtually. The presentation will be available on demand starting at 7:00 AM EDT / 1:00 PM CET on Monday, September 13, 2021.

A webcast of the event will be available for 90 days on the InflaRx website in the Investors section under Events & Presentations.

About InflaRx N.V.:

InflaRx (Nasdaq: IFRX) is a clinical-stage biopharmaceutical company focused on applying its proprietary anti-C5a technology to discover and develop first-in-class, potent and specific inhibitors of C5a. Complement C5a is a powerful inflammatory mediator involved in the progression of a wide variety of autoimmune and other inflammatory diseases. InflaRx was founded in 2007, and the group has offices and subsidiaries in Jena and Munich, Germany, as well as Ann Arbor, MI, USA. For further information please visit
http://www.InflaRx.com.

Contacts:

InflaRx N.V.

Jordan Zwick – Chief Strategy Officer
Jason Stewart – Strategy & IR
Email: [email protected]
Tel: +1 917-338-6523

MC Services AG

Katja Arnold, Laurie Doyle, Andreas Jungfer
Email: [email protected]
Europe: +49 89-210 2280
US: +1-339-832-0752

FORWARD-LOOKING STATEMENTS

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September 9, 2021 7:33 am

Decibel Therapeutics Receives Orphan Drug and Rare Pediatric Disease Designations for DB-OTO for the Treatment of Otoferlin-Related Congenital Hearing Loss

BOSTON, Sept. 09, 2021 (GLOBE NEWSWIRE) — Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s lead gene therapy product candidate, DB-OTO, for the treatment of patients with otoferlin-related congenital hearing loss.

“We are pleased to receive these important designations from the FDA, which support our conviction that innovative treatments for congenital hearing loss are urgently needed,” said Heather Wolff, Vice President, Clinical Development Operations of Decibel. “Preclinical studies support the potential of DB-OTO to provide hearing to children born with profound hearing loss due to a mutation of the otoferlin gene. We are looking forward to initiating a Phase 1/2 clinical trial of DB-OTO in pediatric patients in 2022.”

Orphan Drug Designation is granted to drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases, or conditions affecting fewer than 200,000 people in the United States. The designation affords Decibel the potential for certain benefits, including up to seven years of post-approval market exclusivity, assistance in the drug development process, tax credits for clinical development and exemptions from certain FDA fees.

Rare Pediatric Disease Designation is granted by the FDA to encourage development of treatments for serious or life-threatening rare diseases in which the disease manifestations primarily affect individuals aged from birth to 18 years. Under the Priority Review Voucher program, and subject to FDA approval of DB-OTO for the treatment of otoferlin-related hearing loss, Decibel may be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for a subsequent marketing application for a different product or sold or transferred to another sponsor.

About DB-OTO

DB-OTO is a dual-vector adeno-associated virus (AAV) investigational gene therapy product candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene. The program, developed in collaboration with Regeneron Pharmaceuticals, uses a proprietary, cell-selective promoter to precisely control gene expression in cochlear hair cells. DB-OTO is in preclinical studies, and Decibel expects to initiate clinical testing in 2022.

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel’s pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit www.decibeltx.com or follow us on Twitter.

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